Capricor Therapeutics Inc (CAPR) 2023 Q1 法說會逐字稿

Good afternoon, ladies and gentlemen. This is the conference operator. Welcome to the Capricor first quarter 2023 financial results and corporate update call. (Operator Instructions) As a reminder, this conference call is being recorded. I would now like to turn the conference over to our host, Mr. AJ Bergmann, Capricor's Chief Financial Officer.

女士們、先生們，下午好。這是會議操作員。歡迎參加 Capricor 2023 年第一季度財務業績和公司更新電話會議。 （操作員說明）謹此提醒，本次電話會議正在錄音。現在我想將會議交給我們的東道主 Capricor 首席財務官 AJ Bergmann 先生。

Thank you, and thank you for joining today's call. Before we start, I would like to state that we will be making certain forward-looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety, and intended utilization of our product candidates, our future research and development plans, including our anticipated conduct and timing of preclinical and clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments involving our product candidates, manufacturing capabilities, potential milestone payments, and our possible uses of existing cash and investment resources.

謝謝您，也感謝您參加今天的電話會議。在開始之前，我想聲明，我們將在今天的演示中做出某些前瞻性陳述。這些聲明可能包括有關我們候選產品的功效、安全性和預期用途、我們未來的研究和開發計劃的聲明，包括我們預期的臨床前和臨床研究的進行和時間安排、我們提出或報告額外研究的計劃。數據、我們有關監管備案的計劃、涉及我們候選產品的潛在監管發展、製造能力、潛在的里程碑付款以及我們對現有現金和投資資源的可能使用。

These forward-looking statements are based on current information assumptions and expectations that are subject to change, and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements.

這些前瞻性陳述基於當前的信息假設和預期，可能會發生變化，並涉及許多風險和不確定性，可能導致實際結果與前瞻性陳述中包含的結果存在重大差異。

These and other risks are described in our periodic filings made with the SEC, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements.

這些風險和其他風險在我們向美國證券交易委員會提交的定期文件中進行了描述，包括我們的季度和年度報告。請您注意不要過分依賴這些前瞻性陳述，我們不承擔更新此類陳述的義務。

With that, I turn the call over to Linda Marban, CEO.

說完，我將電話轉給首席執行官琳達·馬班 (Linda Marban)。

Thank you, Ajay. Good afternoon, and thank you for joining our first quarter 2023 conference call. Today, I will provide important updates on our Phase 3 DMD program as well as our exosome platform.

謝謝你，阿傑。下午好，感謝您參加我們的 2023 年第一季度電話會議。今天，我將提供有關我們的 3 期 DMD 計劃以及外泌體平台的重要更新。

We are pleased with the progress that we have made in the first few months of 2023, and believe we are well positioned to execute on our key priorities and reach multiple milestones throughout this year, which include continuing discussions with FDA regarding pathway towards the biologics license application for CAP-1002 in DMD; presenting 24-month follow-up data from our HOPE-2 open label extension study in the second quarter of 2023; reporting the outcome of the interim analysis of HOPE-3 in the fourth quarter of 2023; as well as exploring opportunities for additional strategic partnerships outside of the United States and Japan to support the potential commercialization of CAP-1002 in DMD.

我們對 2023 年前幾個月取得的進展感到高興，並相信我們有能力執行我們的關鍵優先事項並在今年實現多個里程碑，其中包括繼續與 FDA 討論獲得生物製品許可的途徑CAP-1002在DMD中的應用；展示 2023 年第二季度我們的 HOPE-2 開放標籤擴展研究的 24 個月隨訪數據；報告 2023 年第四季度 HOPE-3 中期分析結果；以及探索在美國和日本以外建立更多戰略合作夥伴關係的機會，以支持 CAP-1002 在 DMD 中的潛在商業化。

We also are looking forward to the expansion of our exosome pipeline, focusing on securing partnerships, as well as other opportunities for non-dilutive funding.

我們還期待擴大我們的外泌體管道，重點關注確保合作夥伴關係以及其他非稀釋性融資機會。

Now let me begin my remarks today by providing an update on our recent FDA interactions. Earlier this year, under our RMAT designation, we held a Type B CMC or chemistry, manufacturing, and controls meeting with the FDA where we outlined our plans for produc1tion of commercial scale GMP CAP-1002.

現在，我首先介紹一下我們最近與 FDA 互動的最新情況。今年早些時候，根據我們的 RMAT 指定，我們與 FDA 舉行了 B 型 CMC 或化學、製造和控制會議，會上我們概述了商業規模 GMP CAP-1002 的生產計劃。

We were also able to outline plans for our potency assay and other released criteria in order to support the filing of the BLA, which is a great accomplishment. Although this meeting was focused on CMC aspects of our development program, there was a discussion about the possible needs for some patients to be treated with product manufactured from our GMP San Diego site.

我們還能夠概述我們的效力測定計劃和其他發布的標準，以支持 BLA 的提交，這是一項偉大的成就。儘管這次會議的重點是我們開發計劃的 CMC 方面，但也討論了一些患者可能需要使用我們 GMP 聖地亞哥工廠生產的產品進行治療。

That discussion has not yet been finalized, and is still ongoing. I would like to reiterate that our goal is to work with FDA on the shortest path to filing a BLA. And to that end, we are currently working closely with them on this important issue, and we'll provide updates on our clinical development plan as it becomes available.

該討論尚未最終確定，仍在進行中。我想重申，我們的目標是與 FDA 合作，以最短的路徑提交 BLA。為此，我們目前正在與他們就這一重要問題密切合作，我們將在臨床開發計劃可用時提供最新信息。

This now leads me to an update on our San Diego manufacturing facility for CAP-1002. As you know, we designed this facility within our R&D headquarters to be able to produce commercial-scale GMP CAP-1002 doses. We see this facility as a versatile and cost-effective way to potentially bring CAP-1002 to the market. I am pleased to inform you that we are on track to release GMP CAP-1002 doses in the third quarter of this year.

現在我要了解我們聖地亞哥 CAP-1002 製造工廠的最新情況。如您所知，我們在研發總部設計了這個設施，以便能夠生產商業規模的 GMP CAP-1002 劑量。我們認為該設施是一種多功能且經濟高效的方式，有可能將 CAP-1002 推向市場。我很高興地通知您，我們有望在今年第三季度發布 GMP CAP-1002 劑量。

Should CAP-1002 to obtain market approval, we firmly believe that our ability to manufacture in-house, will greatly increase our margins, and support the early launch of this product. As this facility becomes fully operational, we'll be able to provide more color on potential capabilities and production capacity for this site.

如果CAP-1002獲得市場批准，我們堅信我們的內部製造能力將大大增加我們的利潤，並支持該產品的早期推出。隨著該設施全面投入運營，我們將能夠提供有關該站點潛在能力和生產能力的更多信息。

Next, I would like to provide a clinical update on HOPE-3. Our Phase 3 clinical trial continues to enroll well. As of today, we have 13 active sites, and remain on track to enroll 68 patients by the second half of this year, which is the currently designed sample size.

接下來，我想提供 HOPE-3 的臨床最新進展。我們的 3 期臨床試驗繼續順利入組。截至今天，我們有 13 個活動中心，並有望在今年下半年招募 68 名患者，這是目前設計的樣本量。

We're continuing to activate sites and plan to have additional targeted sites activated by the second quarter, which we believe is both a testament to the high level of engagement by our clinical trial sites as well as our team's strategic execution. Our plans to conduct an interim analysis for sample size re-estimation and analysis of conditional power remains unchanged, and we are on track to have these results available in the fourth quarter of this year.

我們將繼續激活站點，併計劃在第二季度之前激活更多目標站點，我們相信這既證明了我們的臨床試驗站點的高水平參與，也證明了我們團隊的戰略執行力。我們對樣本量重新估計和條件功效分析進行中期分析的計劃保持不變，我們有望在今年第四季度提供這些結果。

As I mentioned previously, we are working closely with the FDA to optimize the HOPE-3 clinical trial designs, and we'll provide updates on any feedback once available should any changes be necessary. In parallel, we continue to treat patients in the open-label extension for OLE portion of the HOPE 2 Phase 2 study. These patients are going to their fifth year of follow-up and going into a third year of OLE treatment.

正如我之前提到的，我們正在與 FDA 密切合作，以優化 HOPE-3 臨床試驗設計，如果有必要進行任何更改，我們將在收到任何反饋後提供更新。與此同時，我們繼續在 HOPE 2 2 期研究的 OLE 開放標籤擴展部分中治療患者。這些患者將進入第五年的隨訪，並進入第三年的 OLE 治療。

Further, while we continue to see efficacy in these patients as previously presented, the safety profile of CAP-1002 in the OLE study continues to be consistent with our Phase 2 results, and is now supported by well over 100 IV infusions.

此外，雖然我們繼續看到之前提出的對這些患者的療效，但 OLE 研究中 CAP-1002 的安全性仍然與我們的 2 期結果一致，並且現在得到了超過 100 次靜脈輸注的支持。

We continue to see a statistically significant as well as clinically relevant slowing down of disease progression for patients treated with CAP-1002, including for these patients who were initially on placebo compared with the natural progression of the disease.

我們繼續看到，與疾病的自然進展相比，接受 CAP-1002 治療的患者（包括那些最初使用安慰劑的患者）疾病進展在統計學上和臨床上均顯著減緩。

The data presented at both the 12 and 18 months showed an average of 65% slowing of disease progression. And we plan to report the 24-month performance of the upper limb and cardiac function data at a medical conference in the second quarter of this year.

第 12 個月和第 18 個月的數據顯示疾病進展平均減緩 65%。我們計劃在今年第二季度的醫學會議上報告上肢24個月的表現和心臟功能數據。

We are thankful to the patients and their families for their continuous commitment to working with us on exploring the potential benefits of CAP-1002. We look forward to building on our body of positive data that further positions CAP-1002 as potential anchor therapy for DMD.

我們感謝患者及其家人持續致力於與我們合作探索 CAP-1002 的潛在益處。我們期待在大量積極數據的基礎上，進一步將 CAP-1002 定位為 DMD 的潛在錨定療法。

Furthermore, we believe the combination therapies may be necessary for the long term to delay disease progression in DMD. Most likely patients requiring a dystrophin replacement therapy will also need additional therapies that can attenuate inflammation, promote healthy muscle growth, and preserve cardiac function.

此外，我們認為，從長遠來看，聯合療法對於延緩 DMD 疾病進展可能是必要的。最有可能需要肌營養不良蛋白替代治療的患者還需要額外的治療，以減輕炎症、促進健康的肌肉生長和保護心臟功能。

We believe that CAP-1002 can potentially be paired with any of the approved therapies, and will likely be necessary to get the greatest benefit from the gene or exon-skipping therapy. We stand with all the patients with DMD whose only wish is that the disease does not get worse.

我們相信 CAP-1002 有可能與任何已批准的療法搭配使用，並且可能是從基因或外顯子跳躍療法中獲得最大益處所必需的。我們與所有 DMD 患者站在一起，他們唯一的願望就是疾病不要惡化。

Now turning to our commercial partnership strategy, as announced in February, we entered into a second agreement with Nippon Shinyaku for the distribution rights to CAP-1002 for DMD in Japan, where we received a $12 million upfront payment, and will potentially receive additional milestone payments of up to approximately $89 million, and a meaningful double digit share of net product revenue.

現在轉向我們的商業合作夥伴戰略，正如 2 月份宣布的那樣，我們與 Nippon Shinyaku 簽訂了第二份協議，獲得用於 DMD 的 CAP-1002 在日本的分銷權，我們收到了 1200 萬美元的預付款，並且可能會獲得額外的里程碑高達約 8900 萬美元的付款，以及淨產品收入中有意義的兩位數份額。

We continue to work with Nippon Shinyaku and look forward to continuing to leverage their expertise and infrastructure already established for Viltepso, their exon-skipping drugs, that is already approved in the US and Japan. We are now focused on securing additional partners in other markets around the world, with Europe being a [clear] priority.

我們將繼續與 Nippon Shinyaku 合作，並期待繼續利用他們為 Viltepso 建立的專業知識和基礎設施，Viltepso 是他們的外顯子跳躍藥物，該藥物已在美國和日本獲得批准。我們現在專注於在全球其他市場尋找更多合作夥伴，其中歐洲是[明確]的優先事項。

Overall, we are delighted with the progress of our DMD program, and we look forward to further sharing updates from our interaction with FDA, our progress with HOPE-3, and the development of potential additional partnerships in new territories.

總的來說，我們對 DMD 項目的進展感到高興，我們期待進一步分享我們與 FDA 互動的最新情況、我們與 HOPE-3 的進展以及在新領域發展潛在的其他合作夥伴關係。

Now briefly turning to our exosome platform technology which leverages the natural cell signaling communication of the body, we are harnessing exosomes to serve as a novel drug delivery system with broad therapeutic applications. Our strong scientific foundation is supportive of further downstream efforts for innovative therapeutic payload loading methods and tissue-specific targeting.

現在簡要介紹一下我們的外泌體平台技術，該技術利用身體的自然細胞信號傳導通訊，我們正在利用外泌體作為具有廣泛治療應用的新型藥物遞送系統。我們強大的科學基礎支持下游創新治療有效負載方法和組織特異性靶向的進一步努力。

Our proprietary StealthX expression platform is at the core of our exosome program and is focused on the development of two broad modalities, vaccinology, and precision therapeutics. We recently published in microbiology spectrum a peer review journal of the American Society of Microbiology on StealthX which in preclinical studies generated two potential vaccine candidates that independently and in combination induced a strong immune response against two SARS-CoV-2 proteins, spike and nucleocapsid.

我們專有的 StealthX 表達平台是我們外泌體項目的核心，專注於疫苗學和精準治療這兩種廣泛模式的開發。我們最近在《微生物學譜系》上發表了美國微生物學會關於 StealthX 的同行評審期刊，該期刊在臨床前研究中產生了兩種潛在的候選疫苗，它們獨立或聯合誘導針對兩種 SARS-CoV-2 蛋白（刺突蛋白和核衣殼）的強烈免疫反應。

Using this StealthX platform, we have successfully developed a targeting strategy which will allow us to potentially expand our exosome program which are precision based therapeutics. While we are exploring many different therapeutic applications, we are currently working on targets in neuromuscular disease which is an area of core strength for Capricor.

利用這個 StealthX 平台，我們成功開發了一種靶向策略，這將使我們能夠潛在地擴展我們的外泌體計劃，即基於精確的治療方法。在我們探索許多不同的治療應用的同時，我們目前正在研究神經肌肉疾病的目標，這是 Capricor 的核心優勢領域。

With our small team of experts working on exosomes, our current plans are to explore business development and partnering strategies as well as non-dilutive grant funding. We look forward to leveraging our exosome platform to support the advancement of next-generation vaccines and innovative targeted therapeutics, and we'll provide updates on this program as they become available.

我們的專家小組致力於外泌體研究，我們目前的計劃是探索業務發展和合作戰略以及非稀釋性贈款資金。我們期待利用我們的外泌體平台來支持下一代疫苗和創新靶向療法的進步，並且我們將在該計劃可用時提供最新信息。

In closing, we are pleased with the advancements across our DMD program and the growing body of data within our exosome platform technology. We look forward to executing on our upcoming milestones. With that, I will turn the call over to Chief Financial Officer, AJ Bergmann, to run through our financial results. AJ?

最後，我們對 DMD 計劃的進步以及外泌體平台技術中不斷增長的數據感到滿意。我們期待著執行即將到來的里程碑。接下來，我將把電話轉給首席財務官 AJ Bergmann，以了解我們的財務業績。阿傑？

Thank you, Linda. This afternoon's press release provided a summary of our first quarter 2023 financials on a GAAP basis. You may also refer to our quarterly report on Form 10-Q which we expect to become available shortly, and will be accessible on the SEC website as well as the financial section of the company website.

謝謝你，琳達。今天下午的新聞稿提供了我們按 GAAP 計算的 2023 年第一季度財務狀況摘要。您還可以參考我們的 10-Q 表格季度報告，我們預計很快就會提供該報告，您可以在 SEC 網站以及公司網站的財務部分訪問該報告。

As of March 31, 2023, the company's cash, cash equivalents, and marketable securities totaled to approximately $45.2 million compared to approximately $41.4 million on December 31, 2022. Based on our current operating plan, the company's cash position is expected to be sufficient to support operations into the fourth quarter of 2024.

截至 2023 年 3 月 31 日，公司的現金、現金等價物和有價證券總計約為 4520 萬美元，而 2022 年 12 月 31 日約為 4140 萬美元。根據我們目前的運營計劃，公司的現金狀況預計足以支持運營至 2024 年第四季度。

I would also like to note that this expectation excludes any potential milestone payments under exclusive commercialization and distribution agreements with Nippon Shinyaku that may become due.

我還想指出的是，這一預期不包括根據與日本新藥簽訂的獨家商業化和分銷協議可能到期的任何潛在里程碑付款。

Turning briefly to the financials, in the first quarter of '23, our net cash provided by operating activities was approximately $4.2 million for the first quarter of 2023. Excluding stock-based compensation, our research and development expense was approximately $7.2 million, compared to approximately $4.9 million in Q1 2022.

簡要地轉向財務數據，2023 年第一季度，我們的經營活動提供的淨現金約為 420 萬美元，2023 年第一季度。不包括股票補償，我們的研發費用約為 720 萬美元，而 2023 年第一季度我們的研發費用約為 720 萬美元。 2022 年第一季度約為 490 萬美元。

Again, excluding stock-based compensation, our general and administrative expenses were approximately $1.8 million in Q1 2023, and approximately $1.9 million in Q1 2022. Net loss for both the first quarter of '23 and '22 was approximately $7.8 million.

同樣，不包括基於股票的薪酬，我們的一般和管理費用在 2023 年第一季度約為 180 萬美元，在 2022 年第一季度約為 190 萬美元。2023 年和 22 年第一季度的淨虧損約為 780 萬美元。

And with that, we will now open the line up for questions. Thank you.

至此，我們現在開始提問。謝謝。

Thank you, AJ.

謝謝你，阿傑。

(Operator Instructions) Joe Pantginis, H.C. Wainwright.

（操作員說明）Joe Pantginis, H.C.溫賴特。

Good afternoon, everyone. This is actually Matt on for Joe. Thanks for taking our questions, and just a couple from us. The first one I have was -- I guess following your conversations with the FDA regarding the Capricor commercialization, are there any additional rate-limiting steps that you guys [heard] or came up with that we should be aware of moving forward?

大家下午好。這實際上是馬特為喬做的。感謝您提出我們的問題，我們僅回答幾個問題。我的第一個問題是——我想在你們與 FDA 關於 Capricor 商業化的對話之後，你們是否[聽到]或提出了我們應該注意的任何額外的速率限制步驟？

Rate-limiting steps in terms of what? In terms of commercialization?

限速步驟是怎樣的？在商業化方面？

Yeah. That's correct. Yeah, yeah.

是的。這是正確的。是啊是啊。

None that we're aware of at this time. We're working closely with FDA, both on the CMC front and also on the clinical development front. And at this point, we feel very encouraged by the attention being paid to CAP-1002 by the agency. They recognize the value of CAP-1002 potentially to patients with DMD. And at this point, we are full speed ahead.

目前我們還不知道。我們正在與 FDA 密切合作，無論是在 CMC 方面還是在臨床開發方面。此時此刻，我們對該機構對 CAP-1002 的關注感到非常鼓舞。他們認識到 CAP-1002 對 DMD 患者的潛在價值。此時此刻，我們正在全速前進。

Okay, perfect. Good to hear. And then my second one is regarding the exosome platform. I know you may not feel to comment, but do you have any additional details or updates regarding the clinical development timeline for that platform?

好的，完美。很高興聽到。我的第二個問題是關於外泌體平台。我知道您可能不想發表評論，但是您是否有關於該平台的臨床開發時間表的任何其他詳細信息或更新？

Yeah. So right now, we're working internally on building that platform therapy. We haven't publicly announced specifically what we're working on. But what I can tell you is that the data is very encouraging in terms of its ability to target the specific cell types as well as the vaccine platform that we recently published in microbiology spectrum.

是的。所以現在，我們正在內部構建該平台療法。我們還沒有公開宣布具體的工作內容。但我可以告訴你的是，就其針對特定細胞類型的能力以及我們最近在微生物學譜中發表的疫苗平台而言，這些數據非常令人鼓舞。

We're exploring, as I mentioned in my prepared remarks, both partnerships and also non-dilutive funding opportunities. What I can tell you is that we believe that this technology will ultimately support the exosome as nature's drug delivery system and its ability to drive biology without toxic consequences.

正如我在準備好的發言中提到的，我們正在探索合作夥伴關係和非稀釋性融資機會。我可以告訴你的是，我們相信這項技術最終將支持外泌體作為自然界的藥物輸送系統，及其驅動生物學而不會產生毒性後果的能力。

Okay. Great. Thanks for taking my questions. I'll go back in the queue.

好的。偉大的。感謝您回答我的問題。我會回到隊列中。

Thanks, Matt.

謝謝，馬特。

Aydan Huseynov, Ladenburg.

艾丹·侯賽諾夫，拉登堡。

Hi, good afternoon, everyone. Linda, AJ, thank you very much for providing updates on the quarter. I have a couple of questions. First, I wanted to start from industry-wide question. I think very soon, probably tomorrow, there will be Sarepta's AdCom meeting about the DMD gene therapy.

嗨，大家下午好。 Linda、AJ，非常感謝您提供本季度的最新情況。我有一些問題。首先，我想從全行業的問題開始。我想很快，可能是明天，Sarepta 將召開有關 DMD 基因治療的 AdCom 會議。

So my question is, do you think there will be any read-across, any spillover effects regarding how we should think about the FDA regulatory path for DMD therapies in general, and how this may affect the CAP-1002?

所以我的問題是，您認為我們應該如何思考 FDA 對 DMD 療法的監管路徑，以及這可能如何影響 CAP-1002，是否會有任何交叉解讀或溢出效應？

Yeah, I think the entire biotechnology industry is going to be impacted by the results of the AdCom. There's a lot of really interesting opportunities there, both for the concept of does it work versus potential patient benefit, and then anecdotal evidence versus actual statistically relevant evidence. All of this is coalescing into sort of a general concept of, what does it mean to get approval? What is necessary for approval? We're all watching that carefully.

是的，我認為整個生物技術行業都會受到 AdCom 結果的影響。那裡有很多非常有趣的機會，無論是對於它是否有效的概念還是潛在的患者利益，以及軼事證據與實際統計相關證據。所有這些都合併成一個一般概念，即獲得批准意味著什麼？審批需要什麼？我們都在仔細觀察。

But I can say specifically about CAP-1002 and DMD is we believe that ultimately the cocktail that will be right for DMD until it can be fixed in utero, will be something to address the dystrophin mutation, but also to deal with the consequences of inflammation and muscle turnover, or the degeneration of the muscle from utilization due to the dystrophin mutation itself.

但我可以具體說的是，關於 CAP-1002 和 DMD，我們相信最終將適合 DMD 的雞尾酒，直到它可以在子宮內修復，這將是解決抗肌營養不良蛋白突變的方法，同時也是應對炎症後果的方法和肌肉更新，或由於肌營養不良蛋白本身突變而導致肌肉無法利用的退化。

So CAP-1002 is being positions really perfectly on combination therapy with any gene therapy or exon-skipping technology because we're going to need that technology to help take care of both the skeletal muscle as well as the cardiac muscle implications. So let me elaborate on that for one more minute.

因此，CAP-1002 非常適合與任何基因療法或外顯子跳躍技術聯合治療，因為我們將需要該技術來幫助照顧骨骼肌和心肌影響。讓我再花一分鐘詳細說明這一點。

Our data which we published in The Lancet of the HOPE-2 clinical trial showed important benefits in ejection fraction which is how the heart meets the needs of the body. What has become clear in a lot of the gene therapy studies is that the heart is not being impacted in a positive way by the gene therapy.

我們在《柳葉刀》上發表的 HOPE-2 臨床試驗數據顯示了射血分數的重要益處，射血分數是心臟滿足身體需求的方式。許多基因治療研究已經明確的是，基因治療並未對心臟產生積極的影響。

So there's going to be a disparate balance of skeletal muscle repair versus cardiac dysfunction, and physicians are talking about this actively right now. It's another way in which CAP-1002 can continue to support and help to sustain quality and perhaps even quantity of life for these boys and young men with DMD.

因此，骨骼肌修復與心臟功能障礙之間將會存在不同的平衡，醫生們現在正在積極討論這個問題。這是 CAP-1002 可以繼續支持和幫助這些患有 DMD 的男孩和年輕人維持生活質量甚至數量的另一種方式。

Thank you, Linda. Appreciate your thoughts. Very helpful. And regarding the breakdown, so I know that you enroll both ambulatory and non-ambulatory patients. Could you provide an update how many you enrolled so far as of today, if you could share that? And is there any way to understand what's the ratio of ambulatory versus non-ambulatory patients so far?

謝謝你，琳達。欣賞你的想法。很有幫助。關於細分，我知道你們招募了門診患者和非門診患者。如果您可以分享的話，您能提供一下截至今天為止您註冊了多少人的最新信息嗎？有什麼方法可以了解迄今為止門診患者與非門診患者的比例是多少？

Yeah. So we're not really quantifying them as ambulatory and non-ambulatory because we're not really stratifying for ambulation. What we're looking for on the inclusion criteria based on the performance of the upper limb ability entry criteria and we're focusing on entry score of 2, which leaves room for decline as well as improvement and an upper-end score of 5, which again leaves room for improvement or decline.

是的。因此，我們並沒有真正將它們量化為步行和非步行，因為我們並沒有真正對步行進行分層。我們正在尋找基於上肢能力准入標準表現的納入標準，我們重點關注准入分數 2，這留下了下降和改進的空間，上限分數為 5，這再次留下了改進或下降的空間。

And so there are many, many people in late stage of ambulation that actually have a significant deficit in the performance of the upper limb. And those are the patients we're focusing on. So we're not looking at ambulation versus non-ambulation in our stratification, but mostly upper limb preservation.

所以有很多很多人在行走後期實際上上肢的功能有明顯的缺陷。這些就是我們關注的患者。因此，我們在分層中關注的不是步行與非步行，而是主要關注上肢的保留。

But I will say we had a really great opportunity to talk recently to a patient with Duchenne muscular dystrophy. His name is Elijah Stacy, and he's published a book called A Small If, if anybody's interested in his story.

但我要說的是，我們最近有一個非常好的機會與杜氏肌營養不良症患者交談。他的名字叫伊利亞·史黛西，如果有人對他的故事感興趣的話，他出版了一本名為《小如果》的書。

But he speaks extremely eloquently about the importance of maintenance of upper limb function, and how many people, including Elijah and his brother are both off their feet but still value their independence to want to maintain upper limb function. So we are laser focused on our patients.

但他極其雄辯地講述了維持上肢功能的重要性，以及有多少人，包括以利亞和他的兄弟，都手足無措，但仍然重視他們的獨立性，想要維持上肢功能。因此，我們非常關注我們的患者。

In terms of enrollments, while we're not disclosing numbers, what I can tell you is enrollment is going very well. We understand from the community and from our sites that families are very interested in CAP-1002, and it makes sense.

在入學人數方面，雖然我們沒有透露具體數字，但我可以告訴你的是，入學進展非常順利。我們從社區和我們的網站了解到，家庭對 CAP-1002 非常感興趣，這是有道理的。

It's a once a quarter infusion that to date has shown to be very safe in patients. And we expect to complete enrollment in the second half of 2023 to the number that we've previously disclosed, the 68 patients, in our randomized, double-blind, placebo-controlled trials HOPE-3.

這是每季度一次的輸注，迄今為止已證明對患者非常安全。我們預計在 2023 年下半年完成隨機、雙盲、安慰劑對照試驗 HOPE-3 中的入組，人數達到我們之前披露的 68 名患者。

Yeah. Yeah, thank you. Yeah, that makes sense in the clinical endpoints, that eventually what is going to matter rather than microdystrophin production. Maybe if you could share, is there any updates on European partnerships, and does Nippon Shinyaku have any appetites to market CAP-1002 in Europe?

是的。是的，謝謝。是的，這在臨床終點上是有意義的，最終重要的是什麼而不是微肌營養不良蛋白的產生。也許您可以分享一下，歐洲合作夥伴關係是否有任何更新，Nippon Shinyaku 是否有興趣在歐洲銷售 CAP-1002？

Our relationship with Nippon Shinyaku is very strong. AJ and I had an opportunity to go visit their headquarters earlier this year, and we were very impressed by their attention to and interest in CAP-1002, both from the US perspective and also from the Japanese perspective.

我們與日本新藥的關係非常牢固。今年早些時候，我和 AJ 有機會參觀了他們的總部，他們對 CAP-1002 的關注和興趣給我們留下了深刻的印象，無論是從美國的角度還是從日本的角度。

We're entertaining all kinds of conversations on the European rights, that we certainly think that they're valuable, and we will select the right partner at the right time, which could possibly be Nippon Shinyaku, but not exclusively looking at them at this time.

我們正在就歐洲權利進行各種對話，我們當然認為它們很有價值，我們會在正確的時間選擇正確的合作夥伴，這可能是日本新藥，但不是專門關注他們時間。

Okay. All right. And the last from me, I'm trying to understand. So are you planning to have any product or indication that you would commercialize on your own without a partner? So if yes, what would that be, product or indication?

好的。好的。最後我想說的，我正在努力理解。那麼，您是否計劃在沒有合作夥伴的情況下自行將任何產品或跡象商業化？如果是的話，那會是什麼，產品還是適應症？

Yeah. So we're constantly evaluating all of these opportunities. Of course, we plan on marketing some on our own as we continue to build CAP-1002. But right now, what we feel is best for the company is that we take it all the way to the door of commercialization and then the appropriate partner, in this case Nippon Shinyaku, for CAP-1002 and DMD has all the infrastructure replaced to move it very quickly, to hopefully a profit generating center.

是的。因此，我們不斷評估所有這些機會。當然，我們計劃在繼續構建 CAP-1002 的過程中自行營銷一些產品。但現在，我們認為對公司來說最好的是我們將其一路推向商業化的大門，然後合適的合作夥伴（在本例中為 Nippon Shinyaku）針對 CAP-1002 和 DMD 更換了所有基礎設施以移動它很快就會成為一個盈利中心。

Okay, Makes sense. Thanks so much for taking my questions. Thank you.

好吧，有道理。非常感謝您回答我的問題。謝謝。

Thanks. Stay well.

謝謝。保持良好。

Thanks, Aydan.

謝謝，艾丹。

(Operator Instructions) This concludes the question and answer session. I would like to turn the conference back over to management for any closing remarks.

（操作員說明）問答環節到此結束。我想將會議轉交管理層發表閉幕詞。

Thank you, operator, and thank you for all who joined us this afternoon and also those who listen later on or read the transcript. We thank all of our patients, all of the families, all of the advocacy groups, and everybody out there who's trying to deal with Duchenne muscular dystrophy.

謝謝接線員，也感謝今天下午加入我們的所有人以及稍後收聽或閱讀文字記錄的人。我們感謝所有患者、所有家庭、所有倡導團體以及所有努力應對杜氏肌營養不良症的人。

And we look forward to providing updates and our attendance at meetings as well as information regarding our progress in the future. Thank you.

我們期待提供最新情況、我們出席會議的情況以及有關我們未來進展的信息。謝謝。

This concludes today's conference call. You may disconnect your lines. Thank you for participating, and have a pleasant day.

今天的電話會議到此結束。您可以斷開線路。感謝您的參與，祝您度過愉快的一天。